FDA Grants Orphan Drug Designation to AUM302 for Neuroblastoma A global clinical-stage biotech company, AUM Biosciences, focused on discovering and developing precision oncology therapeutics, decla...
Gene therapy is an experimental technique that introduces functional genes into a patient’s body to counteract or replace defective ones, thereby curing disease without using pharmaceuticals, radiotherapy, or surgery. Genetic defects that are difficult to treat with drugs or antibodies can be treated with therapy w...Find More
Cutaneous T-cell lymphoma (CTCLs) characterized by cutaneous infiltration of malignant monoclonal T-lymphocytes is a rare lymphoma affecting around 6.4 cases per million individuals every year. The total incident population of Cutaneous T-cell lymphoma in the 7MM was around 7.5K in 2021, with the US accounting for ...Find More
Amgen Reports Promising Mid-stage Results for its Olpasiran Candidate Amgen has reported encouraging mid-stage results for its olpasiran candidate for lowering lipoprotein(a) - a risk factor for atherosclerotic cardiovascular disease - and now plans to move forward with a phase III program as soon as possi...Find More
Hemophilia A treatment scenario before the launch of HEMLIBRA Despite the ups and downs throughout history, the management of hemophilia A patients substantially improved over the past 40 years. The groundbreaking discovery of cryoprecipitate in 1964 marked the beginning of the modern progression of hemophilia t...Find More
In the 21st century, public health has yielded major advances in understanding and managing diseases. Sometimes, early diagnosis remains crucial in managing and treating a serious condition. One such rare genetic disorder, alpha-1 antitrypsin deficiency (AATD), has witnessed improvements concerning awareness and co...Find More
CHMP Gives Positive Opinion on Takeda’s Qdenga The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its approval to Takeda GmbH’s Qdenga, a live, attenuated dengue tetravalent vaccine (TAK-003). It is the first time the CHMP has reviewed a medicinal product ...Find More
Cutaneous T-cell lymphomas (CTCLs) constitute a group of non-Hodgkin lymphomas of the skin. CTCL has an annual age-adjusted incidence of approximately 6 cases per million people. In the United States, approximately 1,000 new cases of skin lymphoma are diagnosed each year. CTCL affects males twice as females. Moreov...Find More
Idiopathic pulmonary fibrosis (IPF) is a rare, sporadic, and fatal interstitial lung disease. As the morbidity and mortality rates associated with IPF remain high, prompt idiopathic pulmonary fibrosis treatment is critical to safeguard individuals’ lung function, reduce the risk of acute exacerbations, and improve ...Find More
AstraZeneca’s Danicopan Shows Positive Results in Phase III Trial Danicopan, an oral Factor D inhibitor developed by AstraZeneca, was expected to fail a phase II trial in rare kidney disease in 2020, but a new readout could revive the drug. Danicopan (ALXN2040) has demonstrated efficacy as an adjunct treatment f...Find More
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